PTC124 Phase 2A Extension Study

This discussion is to hopefully stay connected with other families of boys who participated on the Phase 2a (38 boys in three sites), and to communicate with all the DMD community the progress of our boys while on PTC124.
My son Daniel Castro (10 years old) participated in the 28 days clinical trial of PTC124 along with other 37 boys a couple of years ago. Since, we have been patiently wating for the extension study. Thank God as of last week, we got the confirmation from Dr. Finkel's team in Philadelphia that everything is ready to go, and they are asking ALL the families of the boys who participated to request the weeks in which they would prefer to go to start the new study. If you are one of those families and you haven't gotten in contact with your hospital in which you did the original trial, please contact them ASAP to get this going for our boys. Great news is that our boys will be taken PTC124 for almost 2 years. They will need to get muscle byopsies again, but that is an small price to pay for the potential benefit we all expect to get out of it.
I encourage parents and families in general of these boys to use this discussion to talk about anything related to the development of this new study, and to report progress, complications, experiences, syntoms, differences noticed while in treatment etc. I think it would be good not only for those 38 families, but hopefully for ALL the kids with non-sence mutation who are participating on the new 2b study and even for all other DMD kids with other type of mutations since PTC therapeutics is working on a new medicine for ALL the cases of DMD.
God bless you ALL.
Jose, Angie, Daniel and the Castro Gang.

Replies to This Discussion

Ben was in the original trial in Philly,he goes for his biopsy Jan.7th and will begin taking the drug Jan.21.We saw some results the 1st time and are looking forward to getting back on the drug.Ben is no longer walking and we wonder if this will help him walk again.Tom-Ben's dad.

Hello,
My son Willie in currently involved in Phase 2b located at the Pensacola, Fl site. He has been taking the ptc124/placebo since july. He has experienced some side effects headaches and alot of gas. He has good days and bad but over all he seems to be able to walk longer distances now.
best wishes to all
Martha & Willie

Great Martha, thanks for your input. Since your son is showing minor side effects and/or might be showing some improvement in resistance, it seems he might be taking the medicine. Please keep posting here as you see any changes in him. Best of luck.
As far as our Daniel is concerned, i am very excited as i am typing this email from the Children's hospital of Philadelphia awaiting for tomorrow when my son finally will start taking PTC124 again. I will be paying close attention to any changes on him as well and as i see them i will post them here as well. Best regards, Jose

dacas01We were there the same days. Did you start taking it on the 4th. How is it going. This is our first trial since my son is only 5 1/2. But we are having a hard time getting him to take it any suggestions.

dacas01 said:

Hello all, just an update on my son's status on the trial. We finally are going to Philly next week for the muscle byopsy and are scheduled to go back on Dec 3rd and 4th to start taking the medicine. As we progress in this process i will post here my observations.I am wondering for those who are already on PTC124 for a while... has there been any improvements in energy levels, strenght......? any side effects have been noticed? Could you guys post your observations until now? That would be greatly appreciated by the families behind of you in the process.Thanks

Barb, yes Daniel started taking PTC124 on Dec 4th. He doesn't like it much, but he is 10 years old and he understands better the seriousness of his illnes and what this medicine can mean to him. Therefore he takes it fine. One thing that we are doing is giving him a drink of orange juice or any other juice right after he takes his medicine to help with the flavor and also he likes to get gum afterwards (whatever it takes). Have you tried to give it to him with both milk and water? My son took his first doses with milk, but then he tried with water and he liked it better, so since he is taking it with water. Another thing we are doing is mixing the powder by placing it in an small cup which we can screw the cap (so there is no leake) and shaking it, instead of trying to mix it with an spoon. The first time they gave it to him, they tried to mix it with an spoon and it really doesn't disolve good. When we starting mixing it by shaking it, it disolves better and he likes it better that way. Sorry your son is having a hard time, but try any of the things i just told you (if you haven't) and hopefully any of them will do the trick. Also, as i remember when Daniel participated in the clinical trial IIa about 2.5 years ago, he was having a hard time taking it at the beginning and i started to offer him some rewards (i don't even recall what it was) every time he took his medicine and i only had to do it for a few days because he got used to it. I hope i could help more.
Best Regards
Jose Castro (Daniel's dad)

some news after more than 3 months that my son takes the PTC. I think he takes the medecine and not the placebo because he has had at the beginning side effects but we don' see at the moment any improvment.
has anyone seen improvment?

Hi
My son has been in the trial 9 weeks now,I would not like to guess what he is getting but we think there are improvements specially in energe levels and he feels he can run faster now.We just want to get to end of the trial and then defenately be sure of him being on high dose of ptc 124.Lets hope ptc achieve its purpose.

some news after more than 3 months that my son takes the PTC. I think he takes the medecine and not the placebo because he has had at the beginning side effects but we don' see at the moment any improvment.has anyone seen improvment?